Amyotrophic Lateral Sclerosis | Landscape & Forecast | Disease Landscape & Forecast

Publish date: March 2017

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Amyotrophic lateral sclerosis (ALS) is a degenerative disorder of motor neurons that leads to progressive muscle wasting and weakness, with a typical survival time of three to five years from onset. Riluzole (Concordia Health/Sanofi’s Rilutek, generics) is the only approved disease-modifying therapy (DMT) for ALS, although interviewed experts consider the drug’s efficacy to be modest. Experts emphasize the unmet need for additional, more effective ALS DMTs, as well as new symptomatic drugs, and biomarkers to aid in diagnosis, prognosis, and pharmacodynamics. The ALS clinical pipeline is robust and diverse, comprising late-stage programs aimed at enhancing muscle function, immune modulation, and neuroprotection, as well as mid– or early-stage gene therapy or gene silencing approaches, stem-cell therapies, and immune-targeting agents—and experts contend that regimens combining multiple mechanisms of action could hold significant clinical promise in the management of ALS. Thus, considerable commercial opportunity exists for developers that can overcome the scientific and clinical hurdles blocking the path to regulatory approval for the treatment of this devastating disease.

Questions Answered:

  • Amyotrophic Lateral Sclerosis (ALS) is a rare disease affecting only a small percentage of the population. How large is diagnosed prevalent ALS population in the United States and EU5 (France, Germany, Italy, Spain, and the United Kingdom)? What percentage of the ALS population has a familial versus sporadic form of the disease? What percentage is afflicted with comorbid frontotemporal dementia? How will the size of the ALS population change over the 2016-2026 forecast period? What percentage of diagnosed ALS patients receives drug treatment?
  • With only a single drug approved to prolong survival and maintain function, a high level of unmet need for new ALS treatments exists. What are the key drug targets emerging from basic and clinical research in ALS? What are the most pressing gaps in treatment identified by interviewed ALS experts? How well will emerging therapies in the ALS pipeline address key unmet treatment needs?
  • The ALS pipeline comprises therapies with diverse mechanisms of action and technologies. How are emerging therapies evaluated in clinical trials, and which programs do interviewed experts consider the most promising? Which emerging therapies are likely to launch by 2026, and what clinical and commercial impact will they have?

Scope:

  • Market covered: United States, France, Germany, Italy, Spain, and the United Kingdom.
  • Primary research: Six country-specific interviews with ALS neurologists.
  • Epidemiology: Diagnosed prevalence, diagnosed prevalence by disease type (familial or sporadic ALS), diagnosed prevalence by comorbid frontotemporal dementia.
  • Emerging therapies: Phase III/PR: 3; Phase II: 8; Phase I/II: 2; coverage of select preclinical and Phase I products.
  • Market forecast and alternative market scenarios: Drug-level sales and patient share of ALS therapies in 2026.
  • Key companies: Cytokinetics, Mitsubishi Tanabe Pharma, ​​AB Science, Neuralstem, BrainStorm Therapeutics, Neuraltus Pharmaceuticals, Biogen, Ionis Pharmaceuticals.
  • Key drugs: Tirasemtiv, edaravone, masitinib, riluzole, NSI-566, NurOwn, NP-001.

Table of contents

  • Disease Landscape & Forecast
    • Executive Summary
      • Executive Summary
        • Key Findings
    • Commercial Outlook
      • Market Outlook
        • Key Findings
        • Drivers and Constraints
        • Drug-Class-Specific Trends
        • Alternative Market Scenarios
    • Forecast
    • Etiology and Pathophysiology
      • Etiology and Pathophysiology
        • Disease Overview
        • Expert Insight
      • Etiology
        • Etiology of Amyotrophic Lateral Sclerosis
        • Select Genes Associated with Amyotrophic Lateral Sclerosis
      • Pathophysiology
        • Pathophysiology of Amyotrophic Lateral Sclerosis
      • Disease Presentation
        • Disease Presentation
      • Overlap of Amyotrophic Lateral Sclerosis and Frontotemporal Dementia
      • Disease Burden
      • Prognostic Factors
      • Key Pathways and Drug Targets
    • Epidemiology Overview
      • Introduction
        • Key Findings
        • Expert Insights
      • Epidemiology Populations
        • Diagnosed Prevalent Cases
        • Diagnosed Prevalent Cases by Subtype
        • Diagnosed Prevalent Cases by Comorbid Frontotemporal Dementia
        • Drug-Treated Prevalent Cases
    • Current Treatment Overview
      • Key Findings
        • Key Findings
      • Diagnosis
        • Overview
        • Referral Patterns for Amyotrophic Lateral Sclerosis
      • Treatment Goals
        • Key End Points Used in Clinical Trials for Amyotrophic Lateral Sclerosis
      • Key Current Therapies
        • Riluzole
        • Nuedexta
        • Key Symptomatic Therapies for Amyotrophic Lateral Sclerosis
      • Medical Practice
        • Overview
        • Treatment Guidelines
        • Nonpharmacological Interventions for Amyotrophic Lateral Sclerosis
        • Multidisciplinary Care
        • Region-Specific Treatment
    • Unmet Need Overview
      • Key Findings
        • Key Findings
        • Expert Insight
      • Attainment of Unmet Needs
        • Current Attainment of Unmet Needs in Amyotrophic Lateral Sclerosis
        • Future Attainment of Unmet Needs in Amyotrophic Lateral Sclerosis
    • Emerging Therapies Overview
      • Key Findings
        • Key Findings
        • Expert Insight
        • Pipeline Overview
      • Late-Phase Pipeline Analysis
        • Notable Developments in the Late-Phase Pipeline for Amyotrophic Lateral Sclerosis
        • Muscle Troponin Activators
        • Tyrosine Kinase Inhibitors
        • Neuroprotectants
        • Stem-Cell Therapies
        • Immunomodulators
        • Gene Therapy
        • Combination Therapy for Amyotrophic Lateral Sclerosis
      • Early-Phase Pipeline Analysis
        • Expert Insight: Early-Phase Pipeline
      • Key Discontinuations and Failures in Amyotrophic Lateral Sclerosis
        • Key Discontinuations and Failures in Amyotrophic Lateral Sclerosis
      • Patient Registries
      • Orphan Drug Designation
        • Orphan Drug Provisions: United States
        • Orphan Drug Provisions: Europe
    • Access and Reimbursement Overview
      • Reimbursement of Current Amyotrophic Lateral Sclerosis Therapies
      • Region-Specific Reimbursement Practices
        • United States
        • EU5
    • Methodology
      • Methodology
        • Bottom-Up Market Forecasting Overview
        • Patient Share and Sales Calculations
        • Drug-Treatment Rate Assumptions
        • Patient-Share Assumptions
        • General Sources of Data
        • Dosing, Days of Therapy, and Compliance
        • Price per Treated Day, 2026
    • Appendix
      • Bibliography
      • Experts Interviewed

Author(s): Akash Saini, PhD

Akash Saini, is a lead analyst with the Infectious, Niche, and Rare Disease team at Decision Resources Group. Prior to joining Decision Resources Group, Saini was a postdoctoral fellow at the University of Massachusetts Medical School, where he studied mitochondrial dysfunction in amyotrophic lateral sclerosis (ALS). He earned a in Biochemistry and Biotechnology from the International Centre for Genetic Engineering and Biotechnology, New Delhi, where he also studied the underlying disease mechanism in ALS, and an in Biotechnology from Jawaharlal Nehru University, New Delhi, where he studied the biophysical characteristics of amyloid formation.